Protrans - cell therapy for type 1 diabetes
A research team led by Professor Per-Ola Carlsson is studying the possibility of dampening the immune system attack in type 1 diabetes and preventing further loss of beta cells in the patient's pancreas.
Type 1 diabetes is the result of the body's own immune system being accidentally activated, attacking and killing the beta cells in the pancreas - the cells that produce insulin and regulate blood sugar levels in the body. The affected person only becomes symptomatic when blood sugar levels become high. At this stage, usually 60-80% of the insulin-producing cells have been destroyed and the individual becomes dependent on lifelong treatment with an insulin pump or insulin injections several times a day.
At Uppsala University Hospital, a research team led by Professor Per-Ola Carlsson is studying the possibility of dampening the immune system's attack in type 1 diabetes and preventing further loss of beta cells in the patient's pancreas. Mesenchymal stem cells derived from the umbilical cord and with anti-inflammatory properties are produced and infused into the bloodstream. The treatment aims to maintain the patient's own insulin production, thereby reducing the need for supplemental insulin, improving blood sugar control and ultimately reducing the risk of long-term damage from high blood sugar to blood vessels, kidneys, heart, eyes and other organs.
The treatment has so far been evaluated in clinical trials in adults with newly diagnosed type 1 diabetes. In a first study (Protrans-1; EudraCT No: 2017-002766-50), a phase I part (a safety study) was initially conducted where three different doses of mesenchymal stem cells were evaluated and no side effects of treatment were seen. In a follow-up phase II part of the study, the effect of the treatment was evaluated. Fifteen individuals were randomized to either treatment with stem cells or placebo. One year later, the effect was evaluated and while individuals receiving placebo continued to lose their own insulin production during the year, no significant reduction in insulin production was seen in the patients receiving stem cell treatment. The patients are now being followed for five years after treatment to evaluate both long-term safety and efficacy of treatment. A separate study (EudraCT No:2018-004158-11) has also evaluated the safety and efficacy of repeating treatment with mesenchymal stem cells, Protrans, after one year. Also in these individuals, no side effects of treatment have been seen and with the chosen treatment dose, fully maintained own insulin production is seen even two years after the first treatment.
On the basis of the positive results in the studies on adults with newly diagnosed type 1 diabetes, a treatment study with mesenchymal stem cells in children and adolescents (7-21 years) with newly diagnosed type 1 diabetes (Protrans-YOUNG; EudraCT No: 2020-004520-42) is currently underway at Uppsala University Hospital. Children and adolescents with type 1 diabetes from all over Sweden are included in the study during 2023-2025.
The stem cells used in the treatment come from donated umbilical cords from which the cells are purified and propagated according to ATMP guidelines to be given to diabetic patients.
Foto: The photo depicts, from left to right, Rebecka Hilmius (research nurse), Professor Per-Ola Carlsson (head Physician) and Karin Kjellström (research nurse). Photographer Therese Isaksson.